March 2019 ‘Science Corner’
Check out our March 2019 edition of ‘Science Corner’, with Fernando Vieira, M.D. – the Chief Scientific Officer at ALS Therapy Development Institute. Stay tuned every month for exciting updates on ALS research!
I’m often asked, “What are you most excited about with today’s ALS science? What could be the next breakthrough?”
It’s a tough question to answer, partially because ALS is itself such a complex disease. But one thing that excites and inspires me is how we are partnering with people with ALS to learn how to better combat ALS. And in turn, our scientists are now able to share back valuable, personalized insights with patients about their experience with the disease.
This idea is the cornerstone of our Precision Medicine Program (PMP), of which we’ve now enrolled more than 500 individuals with ALS, and counting. Through the PMP, people with ALS monitor their disease progression in real-time via cutting-edge technologies that detect changes in their disease, particularly their movement and speech, two areas greatly affected by ALS. This individualized information helps people make more informed decisions and better manage their ALS, their progression and response to any interventions. No other research entity actively engages people with ALS in this way, it’s a multifaceted program that is empowering people with ALS.
In an exciting science collaboration with Google, we’re combing through the large datasets collected within the PMP- medical histories, genetics, demographics, voice recordings, and activity trackers. We’re learning ways to trace and define a person’s unique experience with ALS. We believe these approaches can reveal ways to streamline the clinical trial process, making it more agile and less expensive.
One advancement coming out of the PMP that we’re currently exploring in our lab is — utilizing human neural cells “in a dish” to model cellular weakening and death (a prominent characteristic in ALS) to identify potential medicines that could improve the neuron’s energy production and survival.
PMP is an innovative, first-of-its kind model working to drastically speed up the drug validation process for ALS. And, with these crucial data, our scientists are rethinking how we target and treat ALS, uncovering new ideas, likely treatments and pathways to an ultimate cure.
We are committed to growing PMP, enrolling at least 1,000 people with ALS in the program. An ALS breakthrough could come from this robust program, and be one of many important outcomes from our partnership alongside so many living with ALS today.
I hope you’ll encourage the people you love with ALS to reach out and get connected to this program. Please help us spread the word!
Thank you for your generous and continued support of Augie’s Quest – your giving is making our science, the growth and impact of PMP become a reality. You’re helping us get one step closer to realizing the ALS breakthroughs that will change the experience of so many with this devastating disease.
To learn more about the Precision Medicine Program, please visit www.als.net/precision-medicine